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Sickle Cell Disease clinical trials at UCSF
5 in progress, 2 open to new patients

  • Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis

    open to eligible people ages 8–25

    Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the nociceptive system, and as a receptor antagonist, ketamine mitigates this. Intravenous (IV) ketamine has demonstrated efficacy in reducing post-operative, chronic, and cancer-related pain in pediatrics, as well as in reducing time to pain control in the emergency department (ED) in adults. Limited studies suggest efficacy in adult opioid-refractory SCD patients. This study is investigating the safety and tolerability of adjuvant low-dose IV ketamine bolus for pediatric SCD VOE in the ED, as well as its efficacy in improving pain control and reducing hospitalization.

    Oakland, California

  • Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

    open to eligible people ages 2–25

    CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.

    Oakland, California and other locations

  • Dose-Finding Study of SC411 in Children With Sickle Cell Disease

    Sorry, in progress, not accepting new patients

    This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.

    Oakland, California and other locations

  • Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

    Sorry, accepting new patients by invitation only

    This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.

    Oakland, California and other locations

  • Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

    Sorry, in progress, not accepting new patients

    Fludarabine is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and genetic factors cause changes in fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

    San Francisco, California